Abstract:In this series, autologous HSCT was safe and effective in inducing major clinical response and maintained significant benefit for 2 patients at 9 months and 20 months posttreatment, respectively. Sustained response did not occur for 2 of 4 patients. A regimen dose-response effect may exist, but the addition of TBI did not prevent disease relapse for 1 of the patients. More aggressive T cell depletion of the autograft, use of a myeloablative regimen, or use of an allograft may be necessary to decrease relapse r… Show more
“…Although these studies demonstrated good initial response to therapy, they also showed that CR is unusual and serologic or clinical relapse between 12 and 24 months is common. [11][12][13][14] Since the early reports on long-term remissions of RA involved transplantation of allogeneic stem cells, several authors have postulated a graft-versus-autoimmunity effect (GvA) similar to the wellknown-graft-versus-leukemia (GvL) effect in hematological malignancies. [5][6][7] With the recent development of dosereduced conditioning regimens, the issue of allogeneic transplantation in RA became the subject of scientific discussions.…”
Summary:Allogeneic blood stem cell transplantation with reduced conditioning has been proposed as a new, potentially curative treatment option for patients with rheumatoid arthritis (RA). We report a 60-year-old woman with RA and coexisting multiple myeloma who was treated with high-dose melphalan and autologous blood stem cell transplantation (BSCT) followed by a nonmyeloablative allogeneic BSCT from her healthy dizygotic twin brother. She achieved a complete remission of her RA after autologous BSCT, but relapsed early despite complete donor chimerism following successful allogeneic transplantation with reduced intensity conditioning. This case illustrates that allogeneic BSCT following nonmyeloablative conditioning may be an uncertain option for curing patients with RA.
“…Although these studies demonstrated good initial response to therapy, they also showed that CR is unusual and serologic or clinical relapse between 12 and 24 months is common. [11][12][13][14] Since the early reports on long-term remissions of RA involved transplantation of allogeneic stem cells, several authors have postulated a graft-versus-autoimmunity effect (GvA) similar to the wellknown-graft-versus-leukemia (GvL) effect in hematological malignancies. [5][6][7] With the recent development of dosereduced conditioning regimens, the issue of allogeneic transplantation in RA became the subject of scientific discussions.…”
Summary:Allogeneic blood stem cell transplantation with reduced conditioning has been proposed as a new, potentially curative treatment option for patients with rheumatoid arthritis (RA). We report a 60-year-old woman with RA and coexisting multiple myeloma who was treated with high-dose melphalan and autologous blood stem cell transplantation (BSCT) followed by a nonmyeloablative allogeneic BSCT from her healthy dizygotic twin brother. She achieved a complete remission of her RA after autologous BSCT, but relapsed early despite complete donor chimerism following successful allogeneic transplantation with reduced intensity conditioning. This case illustrates that allogeneic BSCT following nonmyeloablative conditioning may be an uncertain option for curing patients with RA.
“…9 In Chicago, four patients with RA have been treated with cyclophosphamide 200 mg/kg, ATG 90 mg/kg, methylprednisolone 3 g followed by a CD34 + cell enriched (2.5-2.7 log T-cell depleted) autograft. 16 Two of the four patients in this trial demonstrated sustained ACR 70 responses, whereas the remaining two were less successful. Likewise two patients in Omaha had ACR 70 responses until 6 months when disease recurrence occurred.…”
Summary:The substantial morbidity and mortality associated with rheumatoid arthritis (RA), while not widely appreciated, provide adequate justification for consideration of highdose immunoablative therapy followed by hematopoietic stem cell transplantation. While some patients with RA follow a benign course, selected subsets of patients have been identified with 5-year survival rates of 40-70%. A number of factors that can be easily determined serve as useful prognostic indicators for poor outcome. These include the presence of many involved joints (total joint count), the degree of functional disability as measured by the health assessment questionnaire and the presence of rheumatoid factor. This article summarises the present status of hematopoietic stem cell transplantation for rheumatoid arthritis and proposes future directions for research.
“…Following HCT, there was a somewhat better response to DMARDs supporting the immunomodulating effect of HCT. There has been variable success of HCT in RA, but the results have not been encouraging as compared to diseases like SSc or MS [87][88][89].…”
Section: Hct In Rheumatoid Arthritis and Juvenile Idiopathic Arthritismentioning
Autoimmune diseases are characterized by recurrent attacks and remissions, but as a rule they progress and eventually cause a severe disability and death. The present chapter contains general characteristics of autoimmune disease pathogenesis, ways to cause immune tolerance by hematopoietic cell transplantation (HCT), clinical aspects of the treatment for established autoimmune diseases with a special attention to multiple sclerosis (MS) and systemic sclerosis (SSc). A profound analysis of authors' point of view and of the available literature has been performed. The promising results allows to consider HCT as a relevant treatment option for a certain autoimmune diseases.
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